Many patients live in the hope that tomorrow will bring a new medicine to treat their debilitating or life-threatening diseases. In recent years, breakthrough medicines have transformed treatment for a wide range of conditions.
From the modern grandchildren of Thorazine and Haldol to the statins that have revolutionized cardiovascular care, these drugs are credited with saving millions of lives.
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Patients suffering from many serious diseases live with the hope that tomorrow will bring them a breakthrough Rucaparib manufacturer drug that improves their condition or saves their lives. America’s biopharmaceutical companies are working hard to meet that need, continuing research and development even after drugs receive FDA approval. These companies have a responsibility to make sure that new medicines are safe and effective. They should not conceal potential risks or overstate their benefits. They should also do everything they can to keep costs down without compromising safety or efficacy.
Breakthrough Drugs
The FDA’s Breakthrough Therapy Program was launched in 2012 with the goal of accelerating the development and approval of drugs to treat serious diseases. Since that time, the agency has approved nearly 140 breakthrough therapies and shortened novel drug approval times by 23%.
The drugs that have received the breakthrough status are characterized by “promising clinical trial evidence, and the potential to benefit patients with unmet medical need,” according to the FDA. The designation has become a magnet for pharmaceutical companies that want to take advantage of the expedited approval process. The drugs receive more attention and marketing, which can lead to misunderstandings and inaccurate claims about their safety and effectiveness.
A recent study analyzed the drug approval data of 396 new medications, including 60 that received the breakthrough status. The researchers compared Cinacalcet manufacturer drugs to similar treatments that weren’t granted the breakthrough designation and looked for differences in trials that supported the drugs’ approval. They found that the clinical trials examining breakthrough medicines “commonly lacked the important features of scientifically rigorous design” such as randomization, double-blinding, and large sample sizes. They also tended to have shorter durations and to enroll fewer participants.
In addition, the researchers discovered that many of the drugs were approved on the basis of surrogate endpoints rather than based on evidence from directly comparing the drug to another treatment option. For example, a breakthrough drug named Leqembi was approved to treat early Alzheimer’s disease based on a study that showed it reduced brain swelling, but not because of its effect on cognitive decline.
The research suggests that breakthrough medicines may be rushed to market without sufficient testing and could result in medication errors or hidden side effects. The FDA’s efforts to accelerate the development and review of drugs should be balanced with a commitment to patient safety, the authors say. “Patients deserve to know the full truth about the strength of the evidence supporting their medicines,” they write. “When the evidence is unclear or skewed, it is vital that physicians and patients be fully informed.” (Bristol-Myers Squibb’s Opdivo is used to treat Hodgkin lymphoma, and Pfizer’s Palbociclib is prescribed for breast cancer.)
Breakthrough Therapies
The development of life-saving Exemestane manufacturer drugs has accelerated in recent years, thanks to innovations such as gene and cell therapies that offer curative treatment for serious diseases. However, the pace of uptake has been slower than expected due to barriers in coverage and reimbursement by commercial and government payers. Until these issues are addressed, patients could miss out on these potentially life-saving therapies.
The FDA’s Breakthrough Therapy Program was established in 2012 to expedite the evaluation of new medicines for a serious disease with preliminary clinical evidence that the drug may demonstrate substantial improvement over existing therapy on one or more clinically significant endpoints. The program has shortened novel drug approval times by about a year and enabled more patients to receive these potentially life-saving treatments.
To analyze the impact of breakthrough status, researchers examined the outcomes of all new cancer therapies approved by the FDA between 2006 and 2018. They compared the PFS gains for drugs that received breakthrough designation with those that did not. The results show that breakthrough therapy drugs on average provide greater health gains and represent better value for money than drugs approved through the traditional pathway.
Breakthrough designation influences clinical trial design by requiring sponsors to meet with FDA officials early and often to ensure the most efficient path to approval. Ideally, sponsors should pursue the designation as early as possible in their development process — ideally prior to the end of Phase II.
Despite the advantages of breakthrough designation, the process still requires a long time to complete. In fact, on average, breakthrough therapies spend five years in the pre-approval phase compared to seven to 12 years for nonbreakthrough therapy. Moreover, most breakthrough therapies are approved on the basis of short, small-scale clinical trials that frequently lack randomization and double-blinding and only include small numbers of patients.
To accelerate the development of novel, life-saving therapies, the FDA and EMA have introduced a number of programs, including the PRIMEExternal Link Disclaimer and the US Breakthrough Therapy Designation programs. Nonetheless, there is a need for further efforts to harmonize their evaluation and review processes to facilitate timely patient access to innovative medicines.
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Supplier Selection
The supply chain is a crucial link in ensuring the timely delivery of life-saving drugs. To achieve this, pharmaceutical companies must focus on developing a comprehensive supplier management strategy that involves methodically selecting and diversifying suppliers, fostering resilient long-term partnerships, instituting robust commodity strategies and risk management practices, and maintaining regulatory compliance to ensure quality.
The Gates Foundation put Prudhomme’s technology on the fast track, hoping to develop a way of delivering his drug at 50 cents a dose for people in the developing world suffering from malaria, tuberculosis, or other diseases with high mortality rates. Earlier this year, the foundation awarded Prudhomme a $1.2 million grant for his efforts.
As governments around the world modernize their regulatory systems to implement different expedited regulatory tools, a shift is occurring in the order of how innovative drugs get to patients. This is most visible in the accelerated approval pathways available in major markets such as the US (FAST TRACK and Breakthrough Designation, BTD), EU (PRIME), China (CHR or PRIority MEdicines) and Japan (pioneering drug designation previously referred to as SAKIGAKE).
This change will require sponsors to rethink their development and regulatory strategies. It also presents opportunities to build business resilience through diversification of sources and suppliers and by investing in innovative technologies.
One recent example of this is an artificial intelligence system developed by Johns Hopkins Medicine that detects sepsis more than six hours sooner than traditional methods — saving hundreds of lives per year. The system, called TREWS (Targeted Real-time Early Warning System), analyzes a patient’s medical records and looks for a number of symptoms that suggest the deadly condition.
Another opportunity for enhancing supply chain resilience lies in fostering collaboration between national regulators to streamline the review process, sharing resources and reducing duplication. A number of initiatives such as Project Orbis, Access consortium and the ASEAN joint assessment have been set up to facilitate these partnerships. This is an important step to creating a globalised regulatory environment, reducing costs and time to market for breakthrough medicines. However, it is important that these collaborative initiatives also consider the specific needs of each region.
